New model unlocks clues to treating deadly childhood cancer

CIRM-funded research at Sanford Burnham Prebys Medical Discovery Institute in San Diego is identifying compounds that could be used to help children battling a deadly brain cancer.

The cancer is choroid plexus carcinoma (CPC), a rare brain tumor that occurs mainly in children. As it grows the tumor can affect nearby parts of the brain resulting in nausea, vomiting and headaches.

Treatment involves surgery to remove the tumor followed by chemotherapy and radiation. However, many of the children are too young to undergo radiation and only around 40 percent are still alive five years after being diagnosed. Even those who do survive often experience life-long consequences such as developmental disabilities.

One obstacle to developing better therapies has been the lack of a good animal model to enhance our understanding of the disease. That’s where this later research, published in the journal Cancer Research, comes in.

The team at Sanford Burnham developed a new mouse model, by knocking out p53, a gene known to suppress tumor formation, and activating a gene called Myc, which is known to cause cancer.  

Robert Wechsler-Reya

In a news release, Robert Wechsler-Reya, the senior author of the paper, says this new model mirrors the way CPC grows and develops in humans.

“This model is a valuable tool that will increase our understanding of the biology of the cancer and allow us to identify and test novel approaches to therapy. This advance brings us one step closer to a future where every child survives—and thrives—after diagnosis with CPC.”

As proof of that the team tested nearly 8,000 compounds against the mouse tumor cells, to see if they could help stop or slow the progression of the disease. They identified three that showed potential of not just stopping the cancer, but of also not harming healthy surrounding cells.

“These compounds are promising, much-needed leads in the quest for an effective CPC treatment,” says Wechsler-Reya. “Our laboratory plans to evaluate these and additional compounds that can effectively treat this cancer.”